For families managing drug-resistant epilepsy, the standard list of options is a familiar and often exhausting one: try another medication, consider surgery, weigh a stimulation device. Each carries its own risks and tradeoffs, and many families have gone through all of them without finding the relief they needed. That backdrop matters for understanding why a therapy currently in clinical trial is drawing real attention from the epilepsy research community.

Early data is showing something that families in this space have been waiting a long time to see.

What NRTX-1001 is doing

The treatment uses a specific type of cell called interneurons, inhibitory nerve cells that produce GABA, the neurotransmitter the brain uses to regulate electrical activity. When the inhibitory signals are disrupted or absent in certain regions, seizures can propagate. NRTX-1001 is designed to restore those signals directly, not by removing tissue or blocking signals with devices, but by introducing cells that can rebuild the function that's missing.

What makes this approach different from surgery isn't just the mechanism. It's what it opens up. Traditional resective surgery requires identifying a single, clear seizure focus and is generally not an option for people with bilateral temporal lobe epilepsy, where seizures originate in both hemispheres. Removing tissue from both sides carries unacceptable cognitive risk. NRTX-1001 is being tested in both bilateral and unilateral cases, which means it could potentially reach people who currently have no surgical path forward.

The numbers families are talking about

Preliminary data from the first two patients in the multicenter trial show more than a 90% reduction in seizure frequency, at follow-up periods of one year and seven months, respectively.

For a family that has spent years counting seizures and adjusting medications and modifying routines to manage unpredictability, that number represents something significant. Not a cure, and not yet a data set large enough to know how broadly it applies. But more than 90% reduction in drug-resistant temporal lobe epilepsy is a result that moves people who follow this research closely. The trial is ongoing, and more patients mean more clarity.

Other advances moving in 2026

NRTX-1001 is the most visible development, but it's not happening in isolation.

Ovid Therapeutics has released positive Phase I results for OV329, a next-generation inhibitor targeting GABA-aminotransferase, the enzyme that breaks down GABA in the brain. A Phase IIa randomized, placebo-controlled trial in adults with drug-resistant focal onset seizures is planned for the second quarter of 2026. Phase I tested safety in healthy volunteers; Phase IIa will test whether it reduces seizures.

Separately, a team across UC San Francisco, UC Santa Cruz, and UC Berkeley has published findings on optogenetics for seizure control. Using a harmless virus to deliver light-sensitive genes to specific neurons, they were able to suppress seizure-like activity in human brain tissue taken directly from epilepsy patients. This is the first time optogenetics has demonstrated control over seizure activity in living human tissue, which matters because it opens the science toward a less invasive alternative to tissue removal. This research is early, but the brain's circuitry intrigues researchers for good reason: understanding how to interrupt the electrical cascade is a different problem than understanding how to remove the region where it starts.

There is also a seizure forecasting trial testing whether patients can receive reliable daily estimates of their upcoming risk, based on patterns identified through intracranial EEG. For many families, the unpredictability of seizures is among the hardest parts of daily life, separate from the seizures themselves. A tool that offers reliable prediction, even without preventing seizures, can change how families plan, prepare, and carry the psychological weight of not knowing.

What this means for your family right now

If your family member is managing drug-resistant epilepsy on current medications or devices that are working, none of this changes what's effective now. What it does change is the landscape of what might become available.

Access to NRTX-1001 and the other trials described here is currently limited to enrolled clinical trial participants. Eligibility for NRTX-1001 typically requires a documented history of drug-resistant epilepsy, a confirmed seizure origin pattern from imaging, and failure of multiple antiseizure medications at adequate doses.

The Epilepsy Foundation's clinical trials database and ClinicalTrials.gov are the most current places to check trial status and eligibility requirements. An epileptologist who specializes in surgical evaluation is most likely to have direct knowledge of which trials may fit a specific patient's profile and whether a referral makes sense.

For families who have been living inside the limits of what treatment can currently offer, these trials represent a genuine expansion of what researchers are attempting and what may eventually be available. That matters, and it's worth knowing about.